Study shows promise of gene therapy for alcohol use disorder

Researchers at Oregon Health & Science University and institutions across the US have found that a form of gene therapy currently used to treat Parkinson’s disease may dramatically reduce alcohol use among chronic heavy drinkers.

Their study in nonhuman primates showed that implanting a specific type of molecule that induces cell growth effectively resets the brain’s dopamine reward pathway in animals predisposed to heavy drinking. The gene therapy procedure involves brain surgery, and may only be useful in the most severe cases of alcohol use disorder. The study is published in the journal Nature Medicine.
The implanted virus is not harmful and carries a gene that codes for the protein known as glial-derived neurotrophic factor, or GDNF. It was injected in a specific area of the brain of a group of rhesus macaque monkeys that voluntarily and heavily drink ethanol diluted in water. After four macaques underwent the procedure, researchers found their consumption dropped by more than 90% compared with a control group.
GDNF is known as a growth factor which enhances the function of neurons in the brain that synthesize dopamine, a feel-good chemical released in the brain. In the case of alcohol use disorder, chronic drinking decreases the release of dopamine. Researchers enhanced dopamine by delivering GDNF to an area of the brain where dopamine is located.
Source: Ford, M.M., George, B.E., Van Laar, V.S. et al. GDNF gene therapy for alcohol use disorder in male non-human primates. Nat Med. 2023. 29:2030–2040.